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A pill to stop the bleeding? new hope for HHT nosebleeds

NCT ID NCT07743671

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Aug 04, 2026 · Last updated Sep 18, 2026 · Updated 5 times

Summary

This phase 3 trial tests whether an experimental oral drug called engasertib can reduce the number of nosebleeds in adults with moderate to severe hereditary hemorrhagic telangiectasia (HHT), a genetic condition that causes abnormal blood vessels and frequent bleeding. About 240 participants will receive either engasertib or a placebo daily for 28 weeks. The main goal is to see if the drug lowers the total number of nosebleed episodes compared to placebo.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
engasertib (also called VAD044), taken as an oral capsule
What this could lead to
If it works, engasertib could become the first approved treatment to reduce frequent nosebleeds in people with HHT, improving daily life and reducing the need for transfusions.
What could go wrong
This is a phase 3 trial, but success is not guaranteed. The drug may not reduce nosebleeds more than a placebo, and side effects are possible. Results will need to be confirmed in this larger study.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 240 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Sep 2026

An estimate. Start dates often move.

Expected to finish

Jun 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Participants are ≥18 years of age at the Screening Visit. 2. Participants have a definite diagnosis of HHT by the Curaçao criteria, defined as spontaneous and recurrent epistaxis and having at least 2 of the following criteria: 1. Multiple telangiectases at characteristic sites: lips, oral cavity, fingers, or nose; 2. Visceral lesions: gastrointestinal telangiectasia and/or pulmonary, hepatic, cerebral, or spinal arteriovenous malformations (AVMs); or 3. A first degree relative with HHT according to these criteria. 3. Participants must have an ESS \>4 at screening, and, in the judgement of the Investigator, participants are expected to have regular epistaxis that typically lasts for several minutes. This criterion is assessed at screening only and does not require reconfirmation prior to randomization on Day 1. 4. Participants have anemia OR in the prior 6 months have received a parenteral infusion of at least 250 mg of iron OR in the prior 6 months have received a red cell or whole blood transfusion. Exclusion Criteria: 1. History or current diagnosis of clinically significant electrocardiogram (ECG) abnormalities. 2. History of significant or uncontrolled skin disorders per Investigator's judgement. 3. Local ablative (eg, cauterization) or surgical procedures on nasal telangiectases \<6 weeks before the Screening Visit. 4. Use of drugs with anti-angiogenic properties, including, but not limited to, bevacizumab, pazopanib, thalidomide, lenalidomide, pomalidomide, tacrolimus, sirolimus, or selective estrogen response modulators (tamoxifen, raloxifene, or bazedoxifene) \< 6 weeks before the Screening Visit. 5. Use of oral tranexamic or epsilon-aminocaproic acid unless they are on a stable dose for at least 4 weeks before the Screening Visit, which will need to be continued during the entire duration of the double-blind Treatment Period.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    4 sites. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Innovative Hematology, Inc.,

    RECRUITING

    Indianapolis, Indiana, 46260, United States

  • Mayo Clinic

    RECRUITING

    Rochester, Minnesota, 55905, United States

  • The University of Chicago Medical Center (UCMC)

    RECRUITING

    Chicago, Illinois, 60637, United States

  • Washington University School of Medicine

    RECRUITING

    St Louis, Missouri, 63110, United States

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